PB1932: GENERIC ELTHROMBOPAG WITH STANDARD IMMUNOSUPPRESSIVE THERAPY IN SEVERE APLASTIC ANEMIA
Authors: C. Ranjit Kumar, A. Aribandi, A. K. K
Journal: HemaSphere (2022)

Background: Acquired aplastic anemia is a result of immune mediated destruction of bone marrow. Triple drug therapy become standard of care in patient who are not having a full matched donor or who are not fit for allogenic stem cell transplantation. Addition of Elthrombopag to standard immunosuppressive therapy improved the response and cure rate, however data with generic Elthrombopag with triple drug therapy is lacking in the literature. In this study we are presenting our data with generic Elthrombopag along with standard immunosuppressive therapy in severe aplastic anemia. Aims: The data for generic Elthrombopag in severe aplastic anemia (SAA) is lacking, we aimed to study the efficacy and safety of generic Elthrombopag in immunosuppressive therapy regimen in SAA. Methods: In this study patient were enrolled with confirmed diagnosis of Severe aplastic anemia who are not having full matched donor [10/10 HLA] or not fit for transplant but eligible for immunosuppressive therapy. Patient were enrolled between march 2020 to December 2021.Enrolled patient were ruled out fanconi anemia and other inherited bone marrow failure syndromes. Total 21 patient were enrolled both pediatric and adults. One patient was non Severe aplastic anemia remaining 20 patient were severe aplastic anemia. All except one received triple drug therapy with standard dosing of Horse ATG at 40mg/kg/day along with Cyclosporine from day 5 and Generic Elthrombopag from day 1 of therapy. Elthrombopag dose was 150 mg daily in patients who were 12 years of age or older, at a dose of 75 mg daily in patients who were 6 to 11 years of age, and at a dose of 2.5 mg per kilogram of body weight per day in patients who were 2 to 5 years of age. Hematological response at 3 were calculated for Absolute Neutrophil count, platelet count. All the data were tabulated and analyzed. Results: Of the total 21 patient enrolled, pediatric age group less than 18(28%) years were 6 and adult age group were 15 (72%). All enrolled patient had bone marrow cellularity <20%. Sex distribution males 15 (72%) and females were 6 (28%). The baseline average hemoglobin was 7.1gm/dl, Average ANC was 430/cmm and Average platelet count was 33,000/cmm. All were transfusion dependent before initiating therapy. ATG dose density was 100% [40mg/kg/day for 4days].Two patient died one 62years old female in less than 4week of therapy due to fungal pneumonia and 2nd case was 19years old male suicide at 7weeks of therapy. Overall hematological response at 3months was 86% with complete remission was 42%. Side effect profile was documented most common adverse event was elevated aminotransferases in 56%,six patient had (28%) had hyperpigmentation of sclera but their bilirubin was normal, abdominal pain in 36%. Summary/Conclusion: Generic Elthrombopag is a potential alternative to innovator molecule for treating the severe aplastic anemia triple drug regimen. In our pilot study we observed similar response rate though it was not a randomized data. Generic molecule would potentially reduces the cost with probable similar outcomes. However due to non randomized data we need to confirm this results with much larger randomized trial with innovator molecule.